Completion of patient enrolment in clinical study of Ruconest® for prophylaxis of hereditary angioedema

Wednesday, January 6, 2016

Pharming Group N.V. announced the successful completion of patient enrolment in the Phase 2 clinical study of RUCONEST®, (recombinant C1 esterase Inhibitor, 50 IU/kg), for prophylaxis in patients with hereditary angioedema (HAE).

Thirty HAE patients deficient in C1 esterase Inhibitor and with a history of at least four attacks per month have been enrolled in the randomized, double-blind study. The patients receive RUCONEST® either once or twice weekly, or placebo in each of three four-week treatment periods. With the crossover design, all patients will receive each of the dosing regimens. The study will evaluate the safety and efficacy of RUCONEST® when used for prophylaxis of angioedema attacks in patients with HAE.

The recruitment phase of the study was initiated in January 2015 and is being conducted at sites in Canada, Europe, Israel and the United States. The trial is being coordinated by principal investigators, Dr. Marco Cicardi, Professor at the University of Milan, and Dr. Marc Riedl, Professor of Medicine and Clinical Director of the US HAEA Angioedema Center at the University of California, San Diego.

“We are pleased with the timely completion of patient enrolment for this double-blind, randomized, placebo-controlled study. We expect the results to be highly informative in determining the safety and efficacy of RUCONEST® to prevent angioedema attacks in patients with HAE. We expect to have the top-line results of the study around the end of the second quarter. If the results are positive, we intend to meet with the FDA and EMA to discuss next steps for the program, including our plans for a new subcutaneous formulation.” said Bruno Giannetti, MD PhD, Chief Operating Officer of Pharming.

Under the terms of the North American licensing agreement with Valeant Pharmaceuticals International, Valeant and Pharming share the development costs for RUCONEST® for prophylaxis of HAE. Pharming will receive an undisclosed milestone payment from Valeant as and when FDA approval for this additional indication is given. RUCONEST® has been granted Orphan Drug designation by FDA for the prophylactic treatment of angioedema caused by hereditary or acquired C1 esterase inhibitor deficiency, with data exclusivity until 2026 under the Biologics Price Competition and Innovation Act.

Important Safety Information for RUCONEST

RUCONEST® is a recombinant C1 esterase inhibitor indicated for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE). Effectiveness in clinical studies was not established in HAE patients with laryngeal attacks.

RUCONEST (C1 esterase inhibitor [recombinant]) is contraindicated in patients with a history of allergy to rabbits or rabbit-derived products, and patients with a history of life-threatening immediate hypersensitivity reactions to C1 esterase inhibitor preparations, including anaphylaxis.

Severe hypersensitivity reactions may occur. The signs and symptoms of hypersensitivity reactions may include hives, generalized urticaria, tightness of the chest, wheezing, hypotension, and/or anaphylaxis during or after injection of RUCONEST. Should symptoms occur, discontinue RUCONEST and administer appropriate treatment. Because hypersensitivity reactions may have symptoms similar to HAE attacks, treatment methods should be carefully considered.

Serious arterial and venous thromboembolic (TE) events have been reported at the recommended dose of plasma-derived C1 esterase inhibitor products in patients with risk factors. Risk factors may include the presence of an implanted venous catheter/access device, prior history of thrombosis, underlying atherosclerosis, use of oral contraceptives or certain androgens, morbid obesity, and immobility. Monitor patients with known risk factors for TE events during and after RUCONEST administration.

RUCONEST has not been studied in pregnant women; therefore, it should only be used during pregnancy if clearly needed.

The most common adverse reactions (incidence ≥2%) were headache, nausea, and diarrhea. The serious adverse reaction in clinical studies of RUCONEST was anaphylaxis.

Please see complete Prescribing Information for RUCONEST.

RUCONEST® (recombinant C1 esterase inhibitor ) is indicated for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE). Effectiveness in clinical studies was not established in HAE patients with laryngeal attacks.

HAE is caused by a deficiency of the C1 esterase inhibitor protein, which is present in blood and helps control inflammation (swelling) and parts of the immune system. A shortage of C1 esterase inhibitor can lead to repeated attacks of swelling, pain in the abdomen, difficulty breathing and other symptoms. RUCONEST contains C1 esterase inhibitor at 50 IU/kg.

When administered at the onset of HAE attack symptoms at the recommended dose, RUCONEST may help to return a patient’s C1 esterase inhibitor levels to normal range and relieve the symptoms of an HAE attack with a low recurrence of symptoms within 24 hours.

RUCONEST is the only recombinant C1 esterase inhibitor approved by the U.S. Food and Drug Administration (FDA) and was approved in July 2014.

Under the Biologics Price Competition and Innovation Act of 2009, RUCONEST was granted Market exclusivity in the USA until July 2026

RUCONEST is designated as an orphan drug by the FDA for the treatment of acute attacks of angioedema caused by hereditary or acquired C1 esterase inhibitor deficiency.

About HAE
Hereditary Angioedema (HAE) is a rare genetic disorder. It is characterised by spontaneous and recurrent episodes of swelling (edema attacks) of the skin in different parts of the body, as well as in the airways and internal organs. Edema of the skin usually affects the extremities, the face, and the genitals. Patients suffering from this kind of edema often withdraw from their social lives because of the disfiguration, discomfort and pain these symptoms may cause. Almost all HAE patients suffer from bouts of severe abdominal pain, nausea, vomiting and diarrhea caused by swelling of the intestinal wall.

Edema of the throat, nose or tongue is particularly dangerous and potentially life-threatening and can lead to obstruction of the airway passages. Although there is currently no known cure for HAE, it is possible to treat the symptoms associated with edema attacks. HAE affects about 1 in 10,000 to 1 in 50,000 people worldwide. Experts believe that a lot of patients are still seeking the right diagnosis: although HAE is (in principle) easy to diagnose, it is frequently identified very late or not discovered at all. The reason HAE is often misdiagnosed is because the symptoms are similar to those of many other common conditions such as allergies or appendicitis. By the time it is diagnosed correctly, the patient has often been through a long-lasting ordeal.

About Pharming Group N.V.
Pharming Group N.V. is developing innovative products for the treatment of unmet medical needs. RUCONEST® (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of angioedema attacks in patients with HAE in the US, Israel, South Korea, all 28 EU countries plus Norway, Iceland and Liechtenstein.

RUCONEST is commercialised by Pharming in Austria, Germany and the Netherlands. RUCONEST is distributed by Swedish Orphan Biovitrum AB (publ) (SS: SOBI) in the other EU countries and in Azerbaijan, Belarus, Georgia, Iceland, Kazakhstan, Liechtenstein, Norway, Russia, Serbia and Ukraine.

RUCONEST is partnered with Valeant Pharmaceuticals International, Inc. (NYSE: VRX/TSX: VRX) in North America, following Valeant’s April 2015 acquisition of Salix Pharmaceuticals Ltd.

RUCONEST is also being evaluated for various additional follow-on indications.

Pharming has a unique GMP-compliant, validated platform for the production of recombinant human proteins that has proven capable of producing industrial volumes of high quality recombinant human protein in a more economical way compared to current cell-based technologies. Leads for Enzyme Replacement Therapy (ERT) in Pompe and Fabry diseases are under early evaluation. The platform is partnered with Shanghai Institute of Pharmaceutical Industry (SIPI), a Sinopharm Company, for joint global development of new products. Pre-clinical development and manufacturing will take place at SIPI and are funded by SIPI. Pharming and SIPI initially plan to utilise this platform for the development of recombinant human Factor VIII for the treatment of Haemophilia A.

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